cabozantinib (Cometriq)

following a full submission considered under the ultra-orphan medicine process

cabozantinib (Cometriq®) is not recommended for use within NHS Scotland.

Indication under review: for the treatment of adult patients with progressive, unresectable locally advanced or metastatic medullary thyroid carcinoma.

In one pivotal, phase III study, cabozantinib was associated with a significant advantage in progression-free survival over placebo. However, the difference between cabozantinib and placebo did not reach statistical significance in the subgroup of patients with Rearranged during Transfection (RET) negative tumours. The summary of product characteristics therefore notes that for patients in whom RET mutation status is unknown or is negative, a possible lower benefit should be taken into account before individual treatment decision.

The submitting company did not present a sufficiently robust economic analysis and in addition their justification of the treatment’s cost in relation to its benefits was not sufficient to gain acceptance by SMC.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.